Talecris Biotherapeutics, Inc. (Nasdaq: TLCR) announced today that it
was granted orphan drug designation by the U.S. Food and Drug
Administration (FDA) for the development of an aerosol formulation of
Alpha1-Proteinase Inhibitor (Human, A1PI) to treat congenital
alpha1-antitrypsin (AAT) deficiency. AAT deficiency is a
chronic, hereditary condition that increases the risk of certain
diseases, especially emphysema, which typically emerges in the fourth
decade of life. Currently, there are no approved, inhaled treatments
available for the treatment of AAT deficiency.
“This orphan drug designation will allow us to move forward
with developing an alternative method of delivering augmentation therapy
for patients who prefer an inhaled mode of administration.”
Orphan drug designation is granted to companies to encourage the
development of treatments that prevent, diagnose or treat rare,
life-threatening or chronic illnesses that affect fewer than 200,000
people per year in the U.S. The designation provides incentives such as
tax credits and potentially seven years of market exclusivity to
companies willing to support the costly research and development
programs associated with developing specialized drugs for a small
population of individuals.
The initiative helps to give patients suffering from rare diseases
access to the same quality of treatment as other patients. Talecris
received a similar orphan drug designation for the aerosolized form of
AAT from the European Commission in June of 2008.